Abstract
Several studies, some of which have been updated during the recent workshop entitled Genome Medicine: Gene Therapy for the Millennium (Rome, 30 September-3 October 2001), have highlighted the usefulness of extrachromosomal or episomal genes in gene targeting strategies. Due to the selectable nature of antibiotic resistance and reporter genes, targeted correction of mutated versions of these extrachromosomal genes allows an accurate quantification of correction frequency. In addition, these model systems facilitate and speed up the optimization of critical parameters for the successful application of gene targeting approaches. In fact, type of cell line, gene delivery system, molar ratio of episomal target/therapeutic constructs, nature and design of therapeutic complexes and different recombinative proteins may be critical for the actual feasibility of each method. Although virus-based approaches are now being investigated as well, this article is focusing on the targeted correction of extrachromosomal genes by the use of small DNA fragments (SDF), chimeric RNA/DNA oligonucleotides (RDO) and triplex-forming oligonucleotides (TFO).
| Original language | English |
|---|---|
| Pages (from-to) | 679-682 |
| Number of pages | 4 |
| Journal | Gene Therapy |
| Volume | 9 |
| Issue number | 11 |
| DOIs | |
| Publication status | Published - 2002 |
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Keywords
- Correction frequency
- Delivery systems
- Extrachromosomal genes
- Gene targeting
ASJC Scopus subject areas
- Genetics
Cite this
Extrachromosomal genes : A powerful tool in gene targeting approaches. / Colosimo, A.; Guida, V.; Palka, G.; Dallapiccola, B.
In: Gene Therapy, Vol. 9, No. 11, 2002, p. 679-682.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Extrachromosomal genes
T2 - A powerful tool in gene targeting approaches
AU - Colosimo, A.
AU - Guida, V.
AU - Palka, G.
AU - Dallapiccola, B.
PY - 2002
Y1 - 2002
N2 - Several studies, some of which have been updated during the recent workshop entitled Genome Medicine: Gene Therapy for the Millennium (Rome, 30 September-3 October 2001), have highlighted the usefulness of extrachromosomal or episomal genes in gene targeting strategies. Due to the selectable nature of antibiotic resistance and reporter genes, targeted correction of mutated versions of these extrachromosomal genes allows an accurate quantification of correction frequency. In addition, these model systems facilitate and speed up the optimization of critical parameters for the successful application of gene targeting approaches. In fact, type of cell line, gene delivery system, molar ratio of episomal target/therapeutic constructs, nature and design of therapeutic complexes and different recombinative proteins may be critical for the actual feasibility of each method. Although virus-based approaches are now being investigated as well, this article is focusing on the targeted correction of extrachromosomal genes by the use of small DNA fragments (SDF), chimeric RNA/DNA oligonucleotides (RDO) and triplex-forming oligonucleotides (TFO).
AB - Several studies, some of which have been updated during the recent workshop entitled Genome Medicine: Gene Therapy for the Millennium (Rome, 30 September-3 October 2001), have highlighted the usefulness of extrachromosomal or episomal genes in gene targeting strategies. Due to the selectable nature of antibiotic resistance and reporter genes, targeted correction of mutated versions of these extrachromosomal genes allows an accurate quantification of correction frequency. In addition, these model systems facilitate and speed up the optimization of critical parameters for the successful application of gene targeting approaches. In fact, type of cell line, gene delivery system, molar ratio of episomal target/therapeutic constructs, nature and design of therapeutic complexes and different recombinative proteins may be critical for the actual feasibility of each method. Although virus-based approaches are now being investigated as well, this article is focusing on the targeted correction of extrachromosomal genes by the use of small DNA fragments (SDF), chimeric RNA/DNA oligonucleotides (RDO) and triplex-forming oligonucleotides (TFO).
KW - Correction frequency
KW - Delivery systems
KW - Extrachromosomal genes
KW - Gene targeting
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U2 - 10.1038/sj.gt.3301749
DO - 10.1038/sj.gt.3301749
M3 - Article
C2 - 12032686
AN - SCOPUS:0036271298
VL - 9
SP - 679
EP - 682
JO - Gene Therapy
JF - Gene Therapy
SN - 0969-7128
IS - 11
ER -